Common Questions

How is a Clinical Trial planned and carried out?

Before a new drug can be tested, an elaborate protocol (or research plan) is written. Depending on who is doing the research it may be written by a pharmaceutical company, government agency, physician, or research group. The protocol includes details about how the treatment will be administered, how patient safety will be maintained, the type of people that would be eligible for the treatment, baseline requirements for participants, the purpose of the study, and the exact procedures that will take place during the study. The protocol must go through a strict regulatory approval process in addition to receiving federal approval. Once a protocol is approved, it can begin the clinical trial process.

Clinical trials go through a series of research phases. The initial phase, also called pre-clinical research, tests new therapies in a laboratory or on animals. This stage of research is designed to obtain preliminary information regarding how long the drug may stay in your system, the effectiveness of the drug, and any potential side effects. From there, promising therapies will enter “clinical research”. Clinical research is then broken down into four phases:

• Phase I - This is the first phase that new treatments are tested in humans. Usually this type of study will involve 20-100 “healthy volunteers”. They are typically confined to a research center for a period of time so that they can be closely observed. During this stage, the treatments are being tested for many of the same things that were tested during the pre-clinical phase. (Initially, however, the dosage levels start much lower than what was used in animals.) Additional preliminary information is obtained regarding the safety of the drug, how your body metabolizes the drug, which dosage method is best (i.e. by mouth, injection, or IV drip), and the effect of eating food with the drug.

• Phase II – Once the initial safety is determined in Phase I, the trial moves into Phase II. In this phase, the drug is tested on a larger group of volunteers (20-300). Researchers assess how effective the drug is and the dosage levels that should be used. When drug development on a new drug fails, this is usually the phase that it happens in.

• Phase III – During this research phase, the experimental drug is compared with the “standard treatment” currently on the market. This phase of the study usually involves multiple research sites and 300-3,000 participants. Although Phase I and II trials typically involve “healthy volunteers”, Phase III trials allow patients to test out new treatment that may be beneficial to them. Typically, a drug must pass at least two Phase III trials in order to be approved to go on the market.

• Phase IV – After a drug/treatment is available on the market, the research does not stop. Phase IV trials are also referred to as “Post Marketing Surveillance Trials”. During this phase, the newly approved treatments are studied for how they interact with other drugs, how different populations (pregnant women, children, cardiac patients) respond to the drug, and are continued to be monitored for safety. If a drug fails safety requirements at this point, it can be pulled off of the market.

Most of the Clinical Trials available at the MVCC include Phase II and Phase III trials. Knowing which phase the research trial is will help you understand the aims of that study. In most cases, the results of these clinical trials are published in scientific or medical journals. If you are interested in seeing the results from the trial you can ask the doctor, nurse, or research coordinator in charge of your treatment. They should be able to help you find the information you are looking for. Keep in mind, however, that it is sometimes many months or even years until the study results are published.